After dogged work by advocates, fifty-five members of Congress have added their signatures to a letter initiated by representatives Zoe Lofgren (D - CA) and Anna Eshoo (D - CA).

The letter urges NIH to respond in a timely fashion to requests for grants. It also asks NIH to report its efforts to fund research as well as the status of specific plans for funding over the next two years.

​Congressional support is crucial for obtaining funding for research because unlike agencies, which are beyond our influence, representatives have an obligation to support the interests of their constituents.____________________

Advocates Obtain Congressional Support for Strengthened ME/CFS Research at NIH

LOS ANGELES, September 9, 2016 – After years of neglect by the National Institutes of Health (NIH), patients suffering from myalgic encephalomyelitis (ME), commonly known as chronic fatigue syndrome (CFS), created a win today as members of Congress came together urging the NIH to do the right thing and strengthen ME/CFS research.

In a formal U.S. House of Representatives letter published today (“the letter”), 55 members of Congress called upon NIH Director Francis Collins to strengthen the NIH’s efforts in ME/CFS biomedical research through a reinvigorated trans-NIH ME/CFS working group as well as additional intramural and extramural research programs.

As the letter explains, “ME/CFS is a complex, debilitating, and chronic disease afflicting 1 to 2.5 million Americans. It costs individuals, the U.S. health care system, and our economy an estimated $17-$24 billion annually. Yet, as the Institute of Medicine noted in its report, ‘Beyond Myalgic Encephalomyelitis/Chronic Fatigue Syndrome: Redefining an Illness,’ there has been ‘remarkably little research funding’ to date to discover its cause or possible treatments.”

Thanks to the hard work of #MEAction, the Solve ME/CFS Initiative (SMCI), and dozens of independent advocates, the letter attracted a broad coalition of bipartisan cosigners led by U.S. Representatives Zoe Lofgren and Anna Eshoo of California. In addition to encouraging advocates all across the country to reach out to their own representatives, SMCI President Carol Head also wrote a personal letter to all 435 representatives, urging them to sign onto the letter.

Said SMCI President Carol Head, “The NIH has failed to live up to its commitment to ME/CFS patients and has not followed the recommendations put forth in the 2015 IOM report; now, thanks to the actions of a coalition of hardworking advocates and members of Congress, we expect this to change.”

The Solve ME/CFS Initiative (SMCI) was founded in 1987 and has established itself as the leading non-profit organization dedicated to ME/CFS. The organization’s mission is to make ME/CFS widely understood, diagnosable, and treatable by stimulating and conducting research aimed at the early detection, objective diagnosis, and effective treatment of ME/CFS. SMCI is the first and only ME/CFS organization to earn the highest possible distinction (a 4-star rating) from Charity Navigator, America’s largest independent charity evaluator.

OnAugust 16, the First Tier Tribunal (UK) ordered the release of the PACE Trial data to Alem Matthees, marking the end of a two-year battle. (You can read the order HERE.)

Mr. Matthees is an Australian researcher, and ME/CFS patient, who has made repeated attempts under the Freedom of Information Act to obtain anonymized data from the PACE trial. Queen Mary University of Londom (QMUL) has managed to quash every request - until now.

In this historic ruling, the tribunal determined that:

1) The information Mr. Matthees requested is not personal, and therefore an exemption based on the possibility that people in the trial could be identified does not apply.

2) Because data are anonymized, invasion of privacy does not apply.

3) There is no indication that the release of anonymized data would discourage future research.

4) There is a strong public interest in releasing the data.

This last point is especially important, as it directly addresses the issue of transparency in research, a topic that has been much in the news lately.

In a 2005 article published in PLoS ONE, Stanford professor John Ionnides claimed that most published research findings were false. There are a number of reasons why research is falsified, including outright plagiarism, conflict of interests (especially true in cases where research is being paid for by pharmaceutical companies), poor methodology, scientific malfeasance, false premises, and general incompetence.

In the case of the PACE trial, conflict of interests led directly to scientific malfeasance. The conflict here stemmed from the unwillingness of NHS to pay for treatment for ME patients. In comparison to treatments such as Ampligen, IVIG, and other immunotherapies, cognitive behavior therapy (CBT) and graded exercise (GET) are relatively cheap to administer.

The PACE trial is not the first trial to make the claim that CBT and GET are beneficial for ME/CFS patients. Trudie Chalder, one of the principals in the PACE study, has been publishing articles since 1989 touting the benefits of CBT and exercise for ME/CFS patients. Nor is she a stranger to faulty methodology as the statistics on some of these studies were questionable.

The PACE trial was the crowning glory to over two decades of research for Chalder, as well as for several other psychiatrists involved in the study. While it is unlikely QMUL will spend any more money on challenging the tribunal's decision, it is equally unlikely that the PACE trial group will abandon its "research" into CBT and GET. In fact, a second PACE study involving adolescents is already under way.

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Press Release from ME Action:

Thursday, 18th August 2016, London, UK - A tribunal has ruled that data from a treatment trial intoChronic Fatigue Syndrome (CFS) must be released, rejecting an appeal from Queen Mary Universityof London (QMUL).

PACE was a £5 million, publicly-funded clinical trial of exercise and cognitive behavioural therapy for CFS. It has been highly influential in determining treatment in the UK and abroad, but has beencontroversial. Academics and patients have both voiced concerns over “misleading” claims. DrRichard Smith, former editor of the British Medical Journal, said in December 2015 of QMUL’s failure to release the data, “…the inevitable conclusion is that they have something to hide”.

QMUL spent over £200,000 on legal fees in this case, to appeal the Information Commissioner’sdecision that they should release anonymised data from the trial. The request for data was madeunder the Freedom of Information Act by Mr Alem Matthees, to allow analysis of the data accordingto the study’s original published protocol.

QMUL made several arguments why the data should not be released, their main claims being thatthe data was personally identifiable information, and was not sufficiently anonymised. However, thetribunal rejected these arguments, noting that QMUL had already shared the data with a smallselection of other scientists, stating, "In our view, they are tacitly acknowledging that anonymizationis effective, or else they would be in breach of the consent agreement and the DPA principles."

The tribunal was satisfied that the data “...has been anonymised to the extent that the risk ofidentification is remote.” The tribunal also noted the "strong public interest in releasing the datagiven the continued academic interest" and "the seeming reluctance for Queen Mary University toengage with other academics they thought were seeking to challenge their findings."

In his correspondence with the court, Mr Matthees expressed “concerns that QMUL are restrictingthe registered researchers to whom they disclose the data upon request.” The tribunal said, “Theevidence before us is not clear but if QMUL are cherry-picking who analyses their data from withinthe recognised scientific research sphere to only sympathetic researchers, there could be legitimateconcerns that they wish to suppress criticism and proper scrutiny of their trial.”

In its submissions QMUL made a number of accusations of harassment from patients, while QMUL’sexpert witness characterized PACE trial critics as "young men, borderline sociopathic orpsychopathic", remarks the Information Commissioner dismissed as "wild speculations".

When pushed to provide evidence of these threats and harassment under cross examination,witnesses speaking for QMUL were unable to do so, and ultimately conceded that "no threats havebeen made either to researchers or participants."

The tribunal found QMUL's assessment of activist behaviour to be, “grossly exaggerated” statingthat “the only actual evidence was that an individual at a seminar had heckled Professor Chalder.”[Professor Chalder is a leading researcher in the PACE trial and a key witness for QMUL.]

Expert reaction to the decision

Jonathan C.W. Edwards, MDEmeritus Professor of MedicineUniversity College London

“I think this is the right decision and I congratulate Mr Matthees on persevering with a veryreasonable request. The report indicates that the Tribunal considered arguments from both sidesvery thoroughly. It has become clear that the reasons given for not providing the informationrequested are essentially groundless. It is also clearly appreciated that critics of the PACE trial arenot young sociopaths - they include senior medical scientists like myself, concerned about poorscience!”

“I am heartened by the Tribunal’s finding that the Commissioner had reached a correct decision inordering release of anonymized data for the PACE trial. The Tribunal’s assessment that theperceived risks of data release were neither substantiated nor demonstrated in the evidence beforethem and that such minimum risk as had been expressed to them would not in their view outweighthe public interest in disclosure of the disputed information is quite important, not only for patientsin this trial and around the world, but also because it underscores how essential transparency andopen, critical review of clinical trials are to the scientific method.”

Keith Geraghty, PhDHonorary Research FellowUniversity of Manchester

"I read the tribunal decision with great interest. I was surprised that the PACE authors declared inevidence that they had shared their trial data with other researchers. I contacted lead author Prof.Peter White to request access to PACE data to run an independent analysis, but my request was firstignored, then later refused. I now understand that the authors shared the data with a select fewacademics who they picked to co-write papers, but they have failed to share the data with thebroader scientific community. Selectively sharing this publicly-funded data with collaborators butrefusing to share data with anyone else, is not in the best interests of patients or science, and itcreates a perception that the PACE team do not want independent critical analysis of this trial. I findit regrettable that the Medical Research Council, who partly funded this very expensive study, didnot specify that the trial data be made available to other researchers.”

This means that there can now be an independent analysis of data from the PACE trial that has beenused to support a number of conclusions and recommendations regarding the benefits of CBT andGET in ME/CFS that are just not consistent with patient evidence for these interventionsHaving attended the hearing, where a number of unsubstantiated and serious accusations weremade against the patient community, I am pleased to see that this 'red herring' was also rejected bythe tribunal. I hope that QMUL will now accept this judgement to release the data and do so without further delay and that they will not spend any more public money on an appeal.”

David Tuller, DrPH, Investigative journalist and public health expertUniversity of California, Berkeley

"This decision is a thorough repudiation of the efforts by the PACE investigators to protect theirclaims and findings from being exposed as utter nonsense. You don't actually need the data todetermine that the trial is a piece of garbage, but having the data at last will make it clear toeveryone. They will likely appeal, but they will ultimately lose."

Alem MattheesPatient and Second RespondentAustralia

I am very pleased with this outcome. Both the Tribunal’s decision and commentary are a longoverdue victory for the patient community, as well as for advocates of clinical trial transparency andopen data sharing. I want to thank everyone who gave support, advice or assistance, as well asanyone who engaged in debate over the PACE trial and the sharing of clinical trial data. This caseended up costing me greatly in time, energy, and health (currently bedridden).

I utilised the FOIA to loosen the vice grip control over the data and allow truly independent and openanalyses that do not rely on the approval of QMUL or the PACE trial investigators. All this cameabout largely because of their refusal to publish or release the protocol-specified outcomes, andtheir generally questionable and poorly or erroneously justified changes to the published trialprotocol, i.e. outcome switching, after the trial was over and/or after seeing trial data. Claims ofclinically significant improvement may be open to interpretation, but false or misleading claims ofrecovery or remission from debilitating illness simply have no place in the scientific literature.

Tom KindlonInformation OfficerIrish ME/CFS Association

I hope Queen Mary University of London won't appeal again and cause more public money andresources to be spent on the case. Now that a court has ruled that the data is non-identifiable andthat releasing it will not contravene agreements with trial participants, there is no good reason tocontinue to withhold it. If QMUL appeal, people may suspect this case was at least partly abouttrying to hide inconvenient results. Indeed, the tribunal decision notice itself raised the question ofwhether QMUL may wish to avoid proper scrutiny of their trial.

Patients want nothing more than to recover from this condition, so misleading claims about recoveryrates are a particularly serious matter. Many are very sceptical of suggestions they can recover withtalk therapy or by steadily increasing their levels of exercise. This is not their experience.

Extraordinary claims require extraordinary evidence but the researchers have not yet released suchevidence: they revised all four aspects of the recovery criteria to make it much, much easier to beclassed as recovered and have so far failed to provide valid justifications for these changes. Some ofthe PACE Trial investigators have conflicts of interest, such as doing work for insurance companies,which can make people concerned about bias.

This is a huge victory for patients, who have a right to examine the evidence for the treatments thataffect their lives. I expect that the recovery rate will only be a small fraction of what the PACEresearchers claimed, due to the dramatic changes they made to the criteria.

Jane ColbyTymes Trust Executive Director

"Tymes Trust is pleased at the judge's ruling. We believe that, pending independent analysis of PACEdata, the MAGENTA (PACEstyle) study in children should be suspended immediately."

Press Release: U.C. San Diego, August 29, 2016. Chronic fatigue syndrome (CFS) is a mysterious and maddening condition, with no cure or known cause. But researchers at the University of California San Diego School of Medicine, using a variety of techniques to identify and assess targeted metabolites in blood plasma, have identified a characteristic chemical signature for the debilitating ailment and an unexpected underlying biology: It is similar to the state of dauer, and other hypometabolic syndromes like caloric restriction, diapause and hibernation.

Dauer is the German word for persistence or long-lived. It is a type of stasis in the development in some invertebrates that is prompted by harsh environmental conditions. The findings are published online in the August 29 issue of PNAS.

“CFS is a very challenging disease,” said first author Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology and director of the Mitochondrial and Metabolic Disease Center at UC San Diego School of Medicine. “It affects multiple systems of the body. Symptoms vary and are common to many other diseases. There is no diagnostic laboratory test. Patients may spend tens of thousands of dollars and years trying to get a correct diagnosis.”

As many as 2.5 million Americans are believed to have CFS. It most often afflicts women in their 30s to 50s, though both genders and all ages can be affected. The primary symptom is severe fatigue lasting at least six months, with corollary symptoms ranging from muscle pain and headaches to sleep and memory problems.

Naviaux and colleagues studied 84 subjects: 45 men and women who met the diagnostic criteria for CFS and 39 matched controls. The researchers targeted 612 metabolites (substances produced by the processes of metabolism) from 63 biochemical pathways in blood plasma. They found that individuals with CFS showed abnormalities in 20 metabolic pathways. Eighty percent of the diagnostic metabolites measured were decreased, consistent with hypometabolic syndrome or reduced metabolism. The diagnostic accuracy rate exceeded 90 percent.

“Despite the heterogeneity of CFS, the diversity of factors that lead to this condition, our findings show that the cellular metabolic response is the same in patients,” said Naviaux. “And interestingly, it’s chemically similar to the dauer state you see in some organisms, which kicks in when environmental stresses trigger a slow-down in metabolism to permit survival under conditions that might otherwise cause cell death. In CFS, this slow-down comes at the cost of long-term pain and disability.”

Naviaux said the findings show that CFS possesses an objectively identifiable chemical signature in both men and women and that targeted metabolomics, which provide direct small molecule information, can provide actionable treatment information. Only 25 percent of the metabolite disturbances found in each person were needed for the diagnosis of CFS. Roughly 75 percent of abnormalities were unique to each individual, which Naviaux said is useful in guiding personalized treatment.

“This work opens a fresh path to both understanding the biology of CFS and, more importantly to patients, a robust, rational way to develop new therapeutics for a disease sorely in need of them.”

The study authors noted additional research using larger groups of participants from diverse geographical areas is needed to validate both the universality and specificity of the findings.

Funding for this research came, in part, from the UC San Diego Christini Fund, The Wright Family Foundation, The Lennox Foundation, the It Takes Guts Foundation, the UC San Diego Mitochondrial Disease Research Fund and gifts from Tom Eames and Tonye Marie Castenada.

At long last, Ampligen is approved somewhere. Not here, unfortunately, but it's a first step. With Argentinian approval, there is the possibility of approval elsewhere.

More to the point, in Argentina at least, there is official acknowledgement that ME/CFS is not a psychological illness.

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Breakthrough Approval Provides Clear Path for Growth in Latin America and the European Union

PHILADELPHIA, Aug. 26, 2016 (GLOBE NEWSWIRE) -- Hemispherx Biopharma, Inc. (NYSE MKT:HEB) (the “Company” or “Hemispherx”), announced that it has received approval of its New Drug Application (NDA) from Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica (ANMAT) for commercial sale of rintatolimod (U.S. tradename: Ampligen®) in the Argentine Republic for the treatment of severe myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). The product will be marketed by GP Pharm, Hemispherx’s commercial partner in Latin America. We believe that rintatolimod is the first drug to receive approval for this indication anywhere in the world. We also believe that there are no other products in the pipeline for approval, worldwide, for this debilitating disease. A copy of the official approval from ANMAT, translated in English, is available on the Company’s website at http://ir.hemispherx.net/Events_Presentations.

The approval was based on submission of two pivotal studies, AMP-502 and AMP-516. Safety data also included additional CFS and non-CFS studies for a total of over 800 subjects including over 100 subjects with severe CFS who received Ampligen® for one year or longer. Several post-approval activities are required to be completed before product launch, including manufacturing site inspections and reimbursement evaluation by the Health Services Authority (SSS), the central health authority in Argentina. “Working closely with our partner in this effort, GP Pharm, our team at Hemispherx addressed all medical and scientific issues presented by ANMAT and deserves great credit for this major success. At Hemispherx, we may be small by big pharma standards, but our commitment to addressing this dire unmet medical need makes us mighty,” stated Hemispherx CEO Tom Equels.

Approval for commercial sale in Argentina provides a platform for potential commercial sales in certain countries within the European Union under regulations that support cross-border pharmaceutical sales of licensed drugs. Hemispherx and GP Pharm are now working to expand the approval of rintatolimod to additional countries with a focus on Latin America. In Europe, approval in a country with a stringent regulatory process in place, such as Argentina, adds further validation for the product as the Early Access Program (EAP) is launched in Europe.

“In Argentina, rintatolimod (Ampligen) has just been commercially approved for the severe disabling form of ME/CFS. The number of patients with ME/CFS is estimated to be over three million worldwide, however, only a portion of these have the severe and disabling form of the disease which we are targeting with this drug,” stated Tom Equels. “Until now, there has been no commercially available effective treatment and there are no advanced clinical candidates, other than rintatolimod, that we are aware of. This commercial approval in Argentina will dramatically improve our ability to treat patients suffering from severe ME/CFS in Latin America. We continue to work aggressively to clarify a path toward approval for those with severe ME/CFS in the United States, where we have Orphan Drug status, and therefore seven years of product exclusivity upon approval. We are greatly encouraged by this new regulatory approval in Argentina. This is the most significant accomplishment to date in Hemispherx’s plan to bring our drug to severe sufferers of ME/CFS worldwide.”

“We have worked diligently with Hemispherx to get to this point, and are now preparing for the commercial launch of rintatolimod for ME/CFS in Argentina,” commented Jorge Braver, chief executive officer of GP Pharm Latin America. “Looking ahead, we will continue to seek approval in additional Latin American countries.”

Author

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About me:I'm a 25-year veteran of CFIDS. I know what it is like to be bedbound for long stretches of time. I also know what it is like to recover, and to relapse. But this blog is not about my personal experience. It is intended to be a resource - a collection of anything that might be helpful to the CFIDS community: book reviews, advice, CFIDS news, research, advocacy, opinion, who's who in our community, fundraising... and occasionally a bit of humor.

Disclaimer: I am not a doctor, which means nothing I write, no matter how sensible it may be, should be interpreted as medical advice.